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Date: 9/11/2017
Drug name:Células madre hematopoyéticas modificadas con un vector lentiviral que contiene el gen CD18 (ITGB2) 
More information: FDA 16-5453
Number EU: 
Description: Deficiencia de adhesión leucocitaria tipo I

Date: 14/10/2016
Drug name: Células madre hematopoyéticas modificadas con un vector lentiviral que contiene el gen CD18 (ITGB2)  
More information: EMA/OD/154/16
Number EU: (EU/3/16/1753)
Description: Deficiencia de adhesión leucocitaria tipo I

Date: 23/06/2016
Drug name:  Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene for the treatment of pyruvate kinase deficiency
More information: FDA designation number: DRU-2016-5168
Number EU: 
Description: Anemia hemolítica por deficiencia de piruvato quinasa de los glóbulos rojos

Date: 02/05/2016
Drug name: Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for the treatment of Fanconi anaemia type A.
More information: FDA Designation request no. 16-5193
Description: Anemia de Fanconi

 

Date: 22/08/2014
Drug name: Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene for the treatment of pyruvate kinase deficiency.
More information: Sponsor CIBERER 
http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/orphans/2014/10/human_orphan_001420.jsp&mid=WC0b01ac058001d12b
Number EU: EU/3/14/1330
Description: http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2014/10/WC500174547.pdf

 

Date: 18/01/2011
Drug name: ORPHA260287: Lentiviral vector carrying the Fanconi anemia-A (FANCA) gene for the treatment of Fanconi anemia type A.
More information: Orphan number: ORPHA260287
Number EU: EU/3/10/822
Description: This orphan designation (EU/3/10/822) was granted by the European Commission to the Centre for Biomedical Network Research on Rare Diseases (CIBERER), Spain, for lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for the treatment of Fanconi anaemia type A.